Oral acromegaly capsule similar to injection therapy in top-line data
An oral form of the somatostatin analogue octreotide was similar to injection therapy as a long-term maintenance treatment for adults with acromegaly, according to top-line data from the MPOWERED trial.
In the phase 3 study, oral octreotide (Mycapssa, Chiasma) demonstrated comparable efficacy to injectable somatostatin analogue injections, meeting the study’s primary noninferiority endpoint. Oral octreotide also maintained mean insulin-like growth factor I within normal limits comparable to injectable therapy, Chiasma stated in a press release.
“These results should provide treating physicians with confidence that patients on injectables who are switched to oral octreotide can be expected to achieve comparable efficacy and safety,” Maria Fleseriu, MD, FACE, professor of medicine and neurological surgery and director of the Pituitary Center at Oregon Health & Science University, lead investigator of the MPOWERED study and an Endocrine Today Editorial Board Member, said in the release. “Many patients with acromegaly experience significant burdens with somatostatin analogue injections, including injection site pain and reactions, and I believe that the results of the MPOWERED study underscore the importance of an oral treatment alternative for patients with acromegaly.”
As Healio previously reported, the FDA approved octreotide in June for long-term maintenance treatment in adults with acromegaly who have responded to and tolerated treatment with injectable octreotide or lanreotide (Somatuline Depot, Ipsen); it is the first and only oral somatostatin analogue approved by the agency.
For MPOWERED, a randomized, open-label and active-controlled trial, researchers analyzed data from 92 adults with acromegaly who were deemed responders to octreotide capsules and completed a 6-month run-in phase. Researchers then randomly assigned participants to octreotide capsules (n = 55) or injectable somatostatin receptor ligands (octreotide long-acting release or lanreotide autogel; n = 37), for an additional 9 months. The trial was designed to evaluate the proportion of participants who maintain their biochemical response to octreotide capsules and patient-reported outcomes in those assigned octreotide capsules compared with those treated with leading injectable somatostatin analogues.
Chiasma reported that the study met its primary noninferiority endpoint, with 91% of participants in the oral octreotide group maintaining IGF-I response (95% CI, 80-97) compared with 100% of participants assigned injectable somatostatin analogue (95% CI, 91-100). Response was defined as the time-weighted average of IGF-I less than 1.3 × upper limit of normal during the 9-month randomized controlled treatment phase.
Participants assigned oral octreotide maintained mean IGF-I levels within normal limits, comparable with injection therapy. Mean IGF-I in the oral octreotide group at the beginning and end of the randomized controlled treatment phase was 0.9 × upper limit of normal and 0.9 × upper limit of normal, respectively, compared with 0.8 × upper limit of normal and 0.8 × upper limit of normal, respectively, in the injectable somatostatin analogue group.
There were no new or unexpected safety signals detected during the study among participants assigned octreotide.
“We are excited with the positive results from the MPOWERED study that demonstrated noninferiority relative to long-acting somatostatin analogues. These results further strengthen the available robust clinical data set for Mycapssa and provide additional meaningful information for health care providers in treating patients with acromegaly,” Raj Kannan, CEO of Chiasma, said in the release. “In light of these positive results, we plan to submit a marketing authorization application for Mycapssa to the European Medicines Agency in mid-2021. While we are excited to report the preliminary top-line results, further analyses of the trial results are ongoing, and we plan to present the full data set from the study at upcoming medical conferences in 2021.”